Verve Therapeutics, on the march toward the clinic with a next-gen heart drug, wants a piece of the ever-growing biotech IPO pie.
Verve debuted just over two years ago with $58.5 million and a goal to bring one-and-done gene editing treatments to heart disease.
Then, in January this year, the company began its march toward the clinic with a treatment for a genetic form of high cholesterol, raising an extra $94 million for its series B and work on the asset.
The treatment, VERVE-101, is a base editor, meaning it doesn’t cut DNA like CRISPR gene editing systems do. Instead, it changes one base, or letter, in the genome to a different one without affecting the letters around it. Its first target? An inherited form of high cholesterol called heterozygous familial hypercholesterolemia, or HeFH.
People with HeFH have a gene mutation in the liver that causes very high cholesterol levels and leads to heart attacks or strokes relatively early in life. VERVE-101 is designed to cut those cholesterol levels by blocking the PCSK9 gene.
With a target and a clinical plan, the young biotech is now seeking a quick turn onto the public markets, gunning for the standard $100 million IPO, though this could swell in the summer heat of biotech IPOs as many have before it.
Verve will use the cash toward its ongoing IND-enabling studies for VERVE-101, with plans to submit an IND next year, according to its SEC-1 filing.
The treatment is a lipid nanoparticle comprising a guide RNA to find the target letter on the PCSK9 and an mRNA that changes an A base in the gene to a G, thus inactivating the gene and lowering cholesterol.
Verve reported data last June showing the approach worked in monkeys. Two weeks after injection, the study found the treatment switched off the PCSK9 gene in 67% of liver cells, causing an 89% drop in PCSK9 protein in the monkeys’ blood and a 59% dip in LDL, or “bad,” cholesterol in their blood. Now, six months down the line, those changes have held.
Verve is starting with HeFH because it’s a serious disease with a clear genetic cause that’s hard to treat. After this first indication, though, Verve plans to branch out into bigger patient populations, first into “the more garden variety” atherosclerotic heart disease and then into the preventive setting.
Verve hopes a one-time gene editing injection could eliminate the hurdles facing cholesterol-lowering statins and other medicines. These include poor adherence, high costs, limited access and lack of insurance, Kathiresan said. And though approval and commercialization are still far out, Verve is already learning how it might roll out its treatments.
As Verve moves VERVE-101 toward the clinic, it will keep plugging along at its preclinical pipeline. Besides PCSK9, it has identified seven other genes, such as ANGPTL3, where loss-of-function mutations—mutations that “break” the gene—protect people from heart attacks. The company is evaluating whether base editing, CRISPR-based editing or an entirely different approach would work best for each target, the biotech told Fierce Biotech at the J.P Morgan annual healthcare conference earlier this year.
It plans to list on the Nasdaq under the ticker "VERV."